Real-world Patient Characteristics Research Articles

44P Real-world (RW) treatment patterns, patient characteristics and outcomes in advanced non-small cell lung cancer (aNSCLC) with PD1/L1 as first-line (1L) therapy in the UK and Germany

The 1L or 2L standard of care (SOC) for patients (pts) with aNSCLC and no targetable genetic alterations [TA] is immunotherapy (IO) with anti-PD-1/L1 agents (PD1/L1) and/or platinum-based chemotherapy (CT). In pts with aNSCLC and EGFR, ALK, or ROS1 alterations, SOC is ≥1 line of tyrosine kinase inhibitors (TKI). The current study aimed to describe RW treatment among pts with PD1/L1 as 1L therapy. This retrospective, observational, two-site cohort study included aNSCLC pts ≥18 years in age who initiated 1L systemic treatment for aNSCLC from 2017 to 2020 from electronic medical records at Leeds Cancer Centre (LCC) in the UK and Frankfurt University Hospital (FUH) in Germany. Pts were analyzed separately at each site with a common protocol and grouped into predefined cohorts: C1 had no TA and 1L PD1/L1; C2 had no TA, 1L+ PD1/L1, and progression; C3 had no TA, 1L+ PD1/L1 ± CT, and progression; C4 had TA, 1L+ TKI(s), and progression; C5 had TA, 1L+ TKI(s) + PD1/L1 + CT, and progression. Only C1 met the evaluable outcome criterion of ≥30 pts per site. The C1 index date was initiation of 1L therapy. At LCC and FUH, C1 included 103 and 104 pts, respectively. C1 was derived from an overall population of 324 (266 with no TA) and 264 (225 with no TA) at LCC and FUH, respectively. C2 to C5 had, as expected, small (unreportable) pt numbers. Most pts in C1 had Stage IV aNSCLC at initial diagnosis and ECOG PS 0 or 1 at start of 1L therapy. In C1 at LCC and FUH, the median (95% CI) RW overall survival (OS) was 15.6 (13.4, 23.3) and 22.2 (16.2, NA) months; RW progression-free survival was 9.0 (6.4, 11.07) and 7.7 (5.4, 12.1) months; at 24 months, the estimated RW OS rate (95% CI) was 36% (27%, 48%) and 48% (37%, 62%).Table: 44PC1 pt characteristics and treatment patternsLCC n = 103FUH n = 104Age, mean, yr66.063.8Male, %54.461.5Stage, %--III21.413.5--IV73.880.8Pathology, %--Squamous17.521.2--Nonsquamous70.971.2ECOG PS, %--016.520.2--162.154.8--2+21.4*21.2**PD-L1 positive, %73.878.8Metastases, %--Brain14.629.8--Visceral54.458.7--Nonvisceral29.146.21L PD1/L1 monotherapy, %53.442.3--TI: 2017–18†§65.8--TI: 2019–20†29.728.81L PD1/L1 + CT ± other anti-cancer, %45.657.7--TI: 2017–18†§34.2--TI: 2019–20†70.371.2§ masked (n <6 for 1L PD1/L1 + CT ± other anti-cancer)* PS 2, 15.5%; PS 3, §; PS 4, §.** PS 2, 16.3%; PS 3, §; PS 4, §.† % in TI periodTI, treatment initiation Open table in a new tab § masked (n <6 for 1L PD1/L1 + CT ± other anti-cancer) * PS 2, 15.5%; PS 3, §; PS 4, §. ** PS 2, 16.3%; PS 3, §; PS 4, §. † % in TI period TI, treatment initiation In this RW study, a larger proportion of pts who initiated 1L treatment in 2019–2020 received PD1/L1 + CT ± other anti-cancer therapy than PD1/L1 monotherapy. Outcomes over time were poor, indicating unmet need.

Real-World Patient Characteristics and Time on Treatment Outcomes Among Patients Receiving First-Line Pembrolizumab in the Treatment of Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC) in the United States

<h3>Purpose/Objective(s)</h3> In July 2019, the FDA approved pembrolizumab in combination with platinum and fluorouracil (P+C), or as monotherapy [P, in combined positive score (CPS) ≥1 population], for the first-line (1L) treatment of patients with R/M HNSCC based on KEYNOTE-048 trial results. Limited evidence exists on the real-world use of pembrolizumab therapy post-approval. The objective of this study was to assess demographic and clinical characteristics, and treatment patterns among patients initiating pembrolizumab therapy in 1L R/M HNSCC. <h3>Materials/Methods</h3> A retrospective cohort study was conducted using the Flatiron Health Advanced Head and Neck database. The study cohort included adult R/M HNSCC patients, who initiated 1L P or P+C between 07/01/2019 - 12/31/2020. Patients were excluded if they received prior platinum treatment ≤6 months of 1L pembrolizumab therapy, had other primary cancers before R/M HNSCC diagnosis, or were treated with a clinical study drug. Real-world time on treatment (rwToT) was assessed using Kaplan-Meier methods. <h3>Results</h3> A total of 402 patients initiated 1L pembrolizumab therapy, [P (n=264), P+C (n=138)]. Descriptive analyses showed differences between patients receiving P versus P+C (Table 1). Patients receiving P were older, and more likely to have a history of smoking, being HPV positive, and have higher ECOG score at 1L initiation. Other patient characteristics were similar between P versus P+C. Treatment pattern results showed that 68.2% (P: 72.0%, P+C: 60.9%) of pembrolizumab treated patients had a record of PD-L1 test before or within 30 days of 1L initiation in the data. Majority of these PD-L1 tests assessed CPS scores (71.8%) with 82.2% of patients CPS≥1 (P 85.8%, P+C 73.2%). The median 1L rwToT was 3.9 months (IQR: 1.4-8.5) for P and 4.9 months (IQR: 1.6−10.4) for P+C, consistent with the results of the KEYNOTE-048 trial [P: 3.5 months (IQR: 1.4-7.6), P+C: 5.8 months (IQR: 2.8-9.7)]. <h3>Conclusion</h3> In our real-world study, some patient differences regarding patient characteristics were observed between patients initiating P vs. P+C. Furthermore, 1L use of pembrolizumab was observed in a severe R/M HNSCC population (ECOG ≥ 2). rwTOT with P and P+C were consistent with results of the KEYNOTE-048 trial, thus validating trial results. Table 1. Baseline characteristics of patients initiating 1L P versus P+C

Characterizing the burden of uncontrolled acromegaly – a description of real-world patient characteristics in Colombia

Objective: To describe the epidemiology and clinical characteristics, relative to biochemical and disease control after treatment initiation, in patients with acromegaly in Colombia. Methods: Retrospective chart review of newly diagnosed acromegaly patients who initiated medical treatment at referral health institutions in Colombia. Patients with controlled vs uncontrolled disease were analyzed, including comorbidities and treatment patterns. In addition, patients receiving somatostatin analogues (SSAs) as first-line treatment (SSA cohort) were compared with those receiving other medical therapy (non-SSA cohort). Results: Data obtained from medical records of 53 patients showed a mean age of 49 years (SD 13.31) and a female preponderance (33 [62.26%]). After 1 year of treatment, 10 (18.9%) patients from the SSA cohort achieved complete biochemical control, showing significantly lower levels of median growth hormone (GH) and insulin-like growth factor-1 (IGF-1) vs 43 (81.1%) patients from the uncontrolled group (p=0.0010 and p=0.0001, respectively). During follow-up, acromegaly-related comorbidities were higher in patients with uncontrolled disease and in the non-SSA group vs the controlled group. Similarly, the percentage of physician visits was lower in the controlled (n=4/10, 40.0%) vs uncontrolled (n=31/43, 72.0%) group and in the SSA (n=24/38, 63.2%) vs non-SSA (n=11/15, 73.3%) cohort. Conclusion: In patients with acromegaly receiving medical therapy, both GH and IGF-1 levels tended to decline over time with the use of SSAs. Lower frequency of comorbidities and use of healthcare resources were associated with controlled acromegaly. Optimizing disease control with adequate patient follow-up and drug treatment may improve clinical outcomes and promote efficient use of healthcare resources.

Real-world patient characteristics associated with survival of 2 years or more after radium-223 treatment for metastatic castration-resistant prostate cancer (EPIX study)

BackgroundThe real-world EPIX study was conducted to gather information about the characteristics of patients with metastatic castration-resistant prostate cancer (mCRPC) who survived ≥2 years after treatment with the alpha-emitter radium-223.MethodsThis retrospective study of electronic health records in the US Flatiron database (NCT04516161) included patients with mCRPC treated with radium-223 between January 2013 and June 2019. Median overall survival (OS) and prostate-specific antigen (PSA) response (≥50% reduction) from start of radium-223 treatment were the primary and secondary endpoints, respectively. Patient characteristics were compared between those who survived ≥2 years versus <2 years, including a subgroup who survived <6 months.ResultsIn the 1180 patients identified, median OS was 12.9 months (95% CI: 12.1–13.7), and 13% of patients with data at 6 months had a PSA response. The survival groups included 775 patients (65.7%) who survived <2 years (including 264 (22.4%) who survived <6 months) and 185 patients (15.7%) who survived ≥2 years; 220 patients (18.6%) had incomplete follow-up data and were censored. On multivariate analysis, age >75 years, Eastern Cooperative Oncology Group performance status (ECOG PS) 2–4, visceral metastases, prior symptomatic skeletal events (SSEs), and prior chemotherapy were independently prognostic of reduced OS. For patients with survival ≥2 years versus <2 years, median age was 71 versus 75 years, 4% versus 14% had ECOG PS 2–4, 4% versus 10% had visceral metastases, 38% versus 44% had prior SSEs, and 16% versus 32% had prior chemotherapy.ConclusionsIn this study of men with mCRPC treated in real-world clinical practice, median OS was consistent with that seen in the phase 3 ALSYMPCA trial. Patients who survived ≥2 years after the start of radium-223 were younger and had better ECOG PS, lower disease burden, and less use of prior chemotherapy than those who survived <2 years.

Abstract P1-18-29: Male patients with hormone receptor positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC) receiving palbociclib in the real-world: patient characteristics, treatment patterns, and outcomes from the POLARIS study

Abstract Background: Although male breast cancer accounts for &amp;lt;1% of all breast cancer diagnoses, its incidence is increasing. Palbociclib in combination with an aromatase inhibitor or fulvestrant is indicated for treating HR+/HER2- ABC in men. Adding to a previous analysis describing real-world patient characteristics and treatment patterns in men with HR+/HER2- ABC, this analysis describes additional patient characteristics, study site characteristics, and preliminary tumor response data in male patients enrolled in the POLARIS study. Methods: POLARIS is a prospective, noninterventional real-world study of patients with HR+/HER2- ABC who received palbociclib as deemed appropriate by a physician; the targeted enrollment is 1500 patients from approximately 110 US and Canadian sites. Patient and study site data were collected from the treating physicians’ routine clinical assessments (eg, patient medical charts, surveys, site questionnaires). Real-world tumor response was determined based on the treating physicians’ assessments of imaging results and clinical progression. Results: As of December 17, 2020, 15 men were enrolled across 12 US sites; most were community-based sites (75%; Table 1) and most physicians (58.3%) had been treating patients with ABC for 11-20 years. The median age of patients was 66 years, 46.7% of patients had visceral disease, and 41.7% had bone-only disease. Seven patients received palbociclib plus an aromatase inhibitor, 7 patients received palbociclib plus fulvestrant, and 1 patient received palbociclib plus tamoxifen. Two patients initiated palbociclib treatment at 100 mg due to comorbidities. Five patients had ≥1 comorbidity (ie, anemia, neutropenia, cellulitis, myocardial infarction, hypertension, neoplasm, or cerebrovascular disorder). All patients received ≥1 concomitant medications (Table 2). Medications commonly received included antihyperglycemics (53.3%), antidepressants (46.7%), opioids (40.0%), antihyperlipidemics (26.7%), and antithrombotics (26.7%). Among all 15 patients, 1 had a best tumor response of complete response (CR) and 4 patients had a partial response (PR), as reported by the physician. Among patients who received palbociclib in the first-line setting (n=9), 1 had a CR and 3 had a PR, as reported by the physician. Conclusions: These findings add to the current knowledge of the male patient population enrolled in the POLARIS study and provide preliminary information on clinical outcomes. This population represents a real-world patient cohort with a heavy disease burden. Although longer follow-up of this patient cohort is warranted, these results continue to support palbociclib as a treatment for men with HR+/HER2- ABC. Pfizer (NCT03280303). Table 1.US Study Site CharacteristicsSite characteristicSites (N=12), n (%)Geographic locationSouth8 (66.7)Midwest3 (25.0)West1 (8.3)Site categoryCommunity9 (75.0)Academic1 (8.3)Other2 (16.7)Number of treating physicians at the practice*Median (range)12 (3-30)Distribution1-104 (33.3)11-205 (41.7)21-302 (16.7)Not reported1 (8.3)Number of breast cancer cases treated annually†Median (range)350 (50, 3000)Distribution50-1502 (16.7)&amp;gt;1508 (66.7)Not reported2 (16.7)Number of HR+/HER2- A/MBC cases treated annually†Median (range)100 (20, 600)Distribution&amp;lt;503 (25.0)50-1503 (25.0)&amp;gt;1504 (33.3)Missing2 (16.7)Use of clinical pathwaysYes7 (58.3)No5 (41.7)A/MBC=advanced/metastatic breast cancer. *Data missing for 1 site; †data missing for 2 sites. Table 2.Documented Concomitant Medications Received in &amp;gt;10% of Patients*MedicationPatients (N=15), n (%)Antihyperglycemics8 (53.3)Metformin4 (26.7)Glimepiride2 (13.3)Antidepressants7 (46.7)Anxiolytics7 (46.7)Alprazolam4 (26.7)Buspirone2 (13.3)Vitamin A and D, including combinations6 (40.0)Cholecalciferol6 (40.0)Opioids6 (40.0)Procet2 (13.3)Calcium3 (20.0)Other analgesics and antipyretics6 (40.0)Paracetamol3 (20.0)Antiemetics and antinauseants5 (33.3)Prochlorperazine3 (20.0)Ondansetron2 (13.3)Antihyperlipidemics4 (26.7)Atorvastatin2 (13.3)Antithrombotics4 (26.7)Rivaroxaban2 (13.3)Peptic ulcer and GORD medications4 (26.7)Pantoprazole2 (13.3)Thyroid medications3 (20.0)Levothyroxine3 (20.0)Angiotensin II receptor blockers3 (20.0)Losartan3 (20.0)ACE inhibitors2 (13.3)Lisinopril2 (13.3)Low-ceiling diuretics, thiazides2 (13.3)Hydrochlorothiazide2 (13.3)High-ceiling diuretics2 (13.3)Furosemide2 (13.3)Vitamin B12 and folic acid2 (13.3)Cyanocobalamin2 (13.3)Hypnotics and sedatives2 (13.3)Zolpidem2 (13.3)ACE=angiotensin-converting enzyme; GORD=gastro-oesophageal reflux disease.*Specific medications received by &amp;gt;10% of patients; information is entered voluntarily and is subject to missing data. This list is not exhaustive. Citation Format: Joanne Blum, Caroline DiCristo, David Gordon, Meghan Karuturi, David Oubre, Erin Jepsen, Juan Cuevas, Shailendra Lakhanpal, Zhe Zhang, Yao Wang, Debu Tripathy. Male patients with hormone receptor positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC) receiving palbociclib in the real-world: patient characteristics, treatment patterns, and outcomes from the POLARIS study [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P1-18-29.

POSC248 Real-World Patient Characteristics and Treatment Patterns in Non-Small Cell Lung Cancer (NSCLC) Patients with KRAS P.G12C Mutant Tumors in Italy

Inhibition of KRAS, and particularly mutant KRAS p.G12C, has been the focus of extensive research in NSCLC but information on how these patients are managed in the real-world setting in Italy is limited. This study aimed to describe the clinical characteristics and treatment patterns in patients with locally advanced and unresectable or metastatic KRAS p.G12C mutant NSCLC in Italy.

Real-world patient characteristics, treatment patterns and costs in relapsing multiple sclerosis patients treated with glatiramer acetate, dimethyl fumarate or teriflunomide in Germany.

Aim: To evaluate adherence, healthcare resource utilization (HRU) and costs for glatiramer acetate (GA; injectable), dimethyl fumarate (oral) and teriflunomide (oral) in relapsing multiple sclerosis. Patients& methods: Retrospective analyses of a claims database. Results: Teriflunomide patients were older with more co-morbidities and fewer relapses versus GA and dimethyl fumarate. GA patients were mostly disease-modifying therapies (DMTs)-treatment naive. Treatment adherence was 61-70%. All DMTs reduced HRU versus pre-index. Costs were comparable across cohorts. High adherence reduced hospitalizations and several costs versus low adherers. Conclusion: Adherence rates were high and comparable with all DMTs. Similar (and high) reductions in HRU and costs occurred with all DMTs. High adherence improved economic outcomes versus low adherence. Thus, investing in adherence improvement is beneficial to improve outcomes in relapsing multiple sclerosis.

Real-World Patient Characteristics and Treatment Patterns of Naldemedine for the Treatment of Opioid-Induced Constipation in Patients with Cancer: A Multicenter Retrospective Chart Review Study.

Background and Objectives: Naldemedine is a peripherally acting μ-opioid receptor antagonist that improves opioid-induced constipation. Although clinical trials have excluded patients with poor performance status (PS) and those started on naldemedine early after opioid initiation, clinical practice has used naldemedine for the same patients. Therefore, we investigated the treatment patterns of naldemedine in a real-world setting. Materials and Methods: This was a multicenter, retrospective chart review study of opioid-treated patients with cancer receiving naldemedine. Adverse events that occurred within 7 days of naldemedine initiation were evaluated in those who received one or more doses of the same. Effectiveness was assessed in patients who used naldemedine for more than 7 days. Results: A total of 296 patients satisfied the eligibility criteria, among whom 129 (43.6%) had a PS of ≥3 and 176 (59.5%) started naldemedine within 2 weeks of opioid initiation. Moreover, 203 (79.6%) patients had ≥3 bowel movements per week. Incidences of all grades of diarrhea and abdominal pain were 87 (29.4%) and 12 (4.1%), respectively. No patient had grade 4 or higher adverse events. Conclusions: Although nearly half of the patients receiving naldemedine in clinical practice belonged to populations that were not included in the clinical trials, our results suggested that naldemedine in clinical practice had the same efficacy and safety as that in clinical trials.

Real-World Patient Characteristics and Treatment Outcomes Among Newly Diagnosed Multiple Myeloma Patients Initiating Daratumumab, Lenalidomide, and Dexamethasone As First-Line Therapy

Introduction: Daratumumab, a CD38 monoclonal antibody, was approved for the treatment of heavily pretreated relapsed or refractory multiple myeloma (MM) in 2015 and for newly diagnosed MM (NDMM) in 2018. The phase 3 MAIA study demonstrated that daratumumab plus lenalidomide/dexamethasone (D-Rd) improved clinical outcomes, including overall survival, versus lenalidomide/dexamethasone (Rd) in transplant-ineligible NDMM (Facon T, et al. EHA Library. 2021). However, limited real-world data are available regarding patients with MM treated with daratumumab in the US. We evaluated patient characteristics and treatment outcomes among transplant-ineligible NDMM patients who received D-Rd as first-line therapy in US oncology practices.Methods: This retrospective, observational cohort study evaluated patients from the Flatiron MM Core Registry who received first-line D-Rd between November 1, 2015 and February 28, 2021. The Flatiron Health electronic health record (EHR)-derived deidentified database has longitudinal patient-level records for patients treated at community oncology practices and academic medical centers across the US. The index date was the date of the first observed record of the D-Rd regimen. Patients aged <18 years on the index date, enrolled in a clinical trial on the index date, with other malignancies prior to the index date, with a diagnosis of amyloid light-chain amyloidosis prior to the index date, or with hematopoietic stem cell transplant from the diagnosis date to the index date were excluded. Patient characteristics analyzed included age at time of D-Rd initiation, race, Eastern Cooperative Oncology Group performance status (ECOG PS) score, International Staging System (ISS) stage, frailty, and presence of comorbidities such as diabetes and acute renal impairment. Time-to-next-treatment and progression-free survival (PFS) were analyzed using the Kaplan-Meier method.Results: From November 1, 2015 to February 28, 2021, 1,721 patients were identified from the Flatiron MM Core Registry as patients treated with a daratumumab-based regimen; 120 (7.0%) of the 1,721 patients were treated with a daratumumab-based regimen as first-line therapy, and 35 (29.2%) of the 120 patients were identified as eligible patients treated with D-Rd as first-line therapy. For patients treated with first-line D-Rd (n=35), the mean age as of the index date was 73.5 years (SD: 7.9). Most patients were White (72.4%), had an ECOG PS score of 1 (51.9%), and had an ISS stage of II (38.1%) or III (42.9%). Of these patients, 15 (42.9%) patients were ≥75 years of age, 4 (11.4%) were Black or African American, 4 (11.4%) had high-risk cytogenetics, 21 (60.0%) were frail, 6 (17.1%) had diabetes, and 6 (17.1%) had acute renal impairment.The median time from the initial diagnosis date to the index date was 1.2 months and the median follow-up was 8.0 months. The median PFS was not reached in this study. The estimated 6-month PFS rate was 91.6%, 9-month PFS rate was 81.0%, and 12-month PFS rate was 73.6% (Figure). After 15 months of follow-up, an estimated 80.5% of patients had not received their next treatment.Conclusions: The real-world population of patients who received D-Rd as first-line therapy is similar to the population studied in MAIA. The early trend of PFS is also similar to that in MAIA, with the majority of patients progression free at 6, 9, and 12 months. Greater understanding of long-term outcomes among transplant-ineligible NDMM patients who received D-Rd as first-line therapy at US oncology practices is needed to further facilitate the safe and effective use of daratumumab. [Display omitted] DisclosuresTai: Janssen Scientific Affairs, LLC: Current Employment. Cai: Janssen: Current Employment. Fu: Janssen: Current Employment. Khare: Janssen: Current Employment. Kaila: Janssen Scientific Affairs, LLC: Current Employment.

Real-World Patient Characteristics, Treatment Patterns and Outcomes for Patients with Atypical Hemolytic Uremic Syndrome Who Have Switched from Eculizumab to Ravulizumab in the USA

IntroductionAtypical hemolytic uremic syndrome (aHUS) is a rare disease that can cause irreversible organ damage or death. Ravulizumab is a long-acting complement C5 inhibitor approved in the USA, Europe, and Japan for the treatment of aHUS. Ravulizumab was engineered from eculizumab to leverage its clinical benefits and safety profile while reducing dosing frequency. This study assessed real-world patient characteristics, treatment patterns, and clinical outcomes for adult and pediatric patients with aHUS in the USA who switched from eculizumab to ravulizumab.MethodsThis retrospective, non-interventional study evaluated the period from January 1, 2012 to March 22, 2021, using US claims data from the Decision Resources Group Real World Data repository. Eligible patients had ≥ 1 medical claim with an aHUS-related International Classification of Diseases 9/10 diagnosis code and ≥ 1 medical or pharmacy claim for treatment with eculizumab or ravulizumab. Patients with evidence of paroxysmal nocturnal hemoglobinuria, myasthenia gravis, neuromyelitis optical spectrum disorder or Shiga toxin Escherichia coli-related hemolytic uremic syndrome in the 3 months prior to their first aHUS-related treatment were excluded. A patient was classified as ‘switched’ if they had ≥ 1 claim for eculizumab followed by an initial claim for ravulizumab from 14 to 30 days after the last eculizumab claim. Patients were required to have ≥ 3 months of continuous enrollment in the database prior to their first ravulizumab claim. Ravulizumab treatment was considered to be continuous if all treatment intervals were ≤ 63 days.Data extracted included: demographics and clinical characteristics at the time of switching to ravulizumab; treatment patterns (including service setting, treatment history before switching to ravulizumab, and subsequent adherence); and clinical outcomes (dialysis, plasma exchange [PE], kidney transplant, and utilization of other end-stage renal disease [ESRD] services) in the 6 months before and after switching to ravulizumab. Data were summarized using descriptive statistics.ResultsOverall, 2101 patients had ≥ 1 eculizumab or ravulizumab claim and an aHUS-related diagnosis. Of these patients, 227 had claims for both eculizumab and ravulizumab with ≥ 3 months of continuous enrollment data before the first ravulizumab claim. The median (lower quartile, upper quartile) treatment interval between the last eculizumab claim and the first ravulizumab claim was 15 (14, 41) days.In total, 131 patients met the ‘switched’ criteria. Table 1 presents patient characteristics at the time of switching from eculizumab to ravulizumab. Outpatient facilities were the most common places of service for initiation of switch to ravulizumab (n = 41/89 patients [46%]); private practice facilities were the most common sites for administration of ravulizumab maintenance treatment (n = 84/213 claims [39%]).Prior to switching to ravulizumab, the median (lower quartile, upper quartile) duration between the first and last eculizumab claim was 788 (252, 1719) days. Among the 95 patients with ≥ 6 months of continuous enrollment after switching to ravulizumab, 73 (77%) patients were treated with ravulizumab continuously for ≥ 6 months. Selected clinical outcomes in the 6 months before and after switching to ravulizumab treatment are presented in Table 2. Although no inferential statistical comparisons were made, data show that fewer switched patients underwent dialysis or utilized other ESRD services in the 6-month post-switch period (with no recorded cases of PE or kidney transplant) compared with the 6-month pre-switch period.Discussion and conclusionsThis study provides real-world data on clinical characteristics and treatment patterns for patients with aHUS who have switched from eculizumab to ravulizumab. The majority of patients switching to ravulizumab were treated continuously for ≥ 6 months according to the treatment schedule in the prescribing information. Ongoing analyses with longer follow-up time will provide inferential assessment of the clinical and economic impacts of switching to ravulizumab. [Display omitted] DisclosuresWang: Alexion: Current Employment; AstraZeneca: Current Employment. Al-Dakkak: Alexion: Current Employment; AstraZeneca: Current Employment. Garlo: Alexion: Current Employment; AstraZeneca: Current Employment. Ong: Alexion: Current Employment; AstraZeneca: Current Employment. Tomazos: Alexion, AstraZeneca Rare Disease: Current Employment. Mahajerin: Alexion: Consultancy; AstraZeneca: Current Employment.

Tumor Lysis Syndrome: Incidence, Mitigation and Management in Patients with Chronic Lymphocytic Leukemia Initiating Venetoclax in Routine Clinical Practice (DEVOTE) across Canada

Tumor Lysis Syndrome: Incidence, Mitigation and Management in Patients with Chronic Lymphocytic Leukemia Initiating Venetoclax in Routine Clinical Practice (DEVOTE) across Canada

P-167: Real-world elderly myeloma patients: improved survival despite more adverse risk factors than younger patients and RCT populations. A study on behalf of the Nordic Myeloma Study Group

<h3>Background</h3> Elderly multiple myeloma (MM) patients are underrepresented in randomized clinical trials (RCTs). Prospective registries provide insight into real-world patient characteristics, treatment and outcome. The Danish Multiple Myeloma Registry (DMMR) and the Swedish Myeloma Registry (SMR), established in 2005 and 2008 respectively, are nationwide prospective registries with near 100% coverage. <h3>Methods</h3> We describe baseline characteristics, treatment and survival for patients diagnosed with active myeloma in the DMMR January 1st 2005-February 18th 2020, and the SMR January 1st 2008-December 31st 2019. We performed a retrospective comparison of patients aged ≥75 years at diagnosis to MM patients <75 years. Further, we compared our cohort to the populations in pivotal RCTs guiding the treatment of elderly myeloma patients. <h3>Results</h3> In total, we report on 4647 Swedish and Danish MM patients ≥75 years at diagnosis compared with MM patients <75 years (n=7378). Out of 4691 newly diagnosed MM patients in the DMMR, 36% (n=1688) were ≥75 years. Among 7334 MM patients in the SMR, 40% (n=2959) were ≥75 years. The elderly cohort presented with more advanced disease (46% ISS III in both registries vs 30.3% (SMR) and 34.6% (DMMR) among the younger cohorts). The proportion of patients with anemia and/or renal failure was higher in the older cohort. In comparison, important RCTs underpinning current treatment guidelines included a lower proportion of elderly patients and patients with ISS III. 30% of patients in the VISTA-trial (VMP vs MP) and the ALCYONE-trial (Dara-VMP vs VMP) were ≥75 years. In the FIRST trial (Rd continuously vs Rd 18 months vs MPT) 35% of patients were >75 years, while the MAIA trial (Dara-Rd vs Rd) has the largest proportion of elderly patients among these studies (43.6%). However, in the MAIA trial only 29% of patients presented with ISS III, compared to 46% of patients ≥75 years in our real-world population. Similarly, 35% of patients in VISTA and 38% in ALCYONE had ISS III. The FIRST trial had a composition more similar to the Swedish and Danish registry population with 35% of patients >75 years, and 48% of these patients with ISS stage III. The treatment strategies in the elderly were similar in Denmark and Sweden. Melphalan and prednisolone (MP) were replaced by bortezomib-based regimes from around 2012, while lenalidomide-based treatment increased in recent years. Median relative survival (RS) for patients ≥75 years in Denmark increased from 25 months to 36 months for patients diagnosed 2005-2007 and 2015-2016, respectively. Similarly, in Sweden the median RS increased from 24 months to 42 months for patients ≥75 years diagnosed 2008-2009 and 2016-2017, respectively. <h3>Conclusion</h3> The real-world MM population is older and has a higher proportion of patients with ISS III disease than patients included in the pivotal RCTs, and compared to the younger patient cohort. Future studies in MM patients should take this into account.

PD-0740 Real-world patient & treatment characteristics of oligometastatic disease: results of OligoCare

PD-0740 Real-world patient & treatment characteristics of oligometastatic disease: results of OligoCare

Patient characteristics, treatment patterns and clinical outcomes among patients with previously treated recurrent or metastatic cervical cancer: a community oncology-based real-world analysis

<h3>Objectives:</h3> The GOG240 trial introduced a doublet chemotherapy with bevacizumab as the new standard of care for first-line (1L) recurrent or metastatic cervical cancer (r/mCC). While this regimen has been shown to prolong survival compared with chemotherapy alone in this advanced population, there is no standard systemic treatment for r/mCC after failure of 1L. The objectives of this study were to characterize the real-world patient characteristics, treatment patterns, and clinical outcomes of patients who initiated second-line (2L) therapy for r/mCC in the US community oncology setting. <h3>Methods:</h3> This was an observational study of women with r/mCC who initiated 2L systemic therapy within the US Oncology Network (USON) between 2014 and 2019. USON's electronic health record was used to identify eligible patients and abstract data. Overall survival (OS), time to treatment discontinuation (TTD), and time to first subsequent treatment (TFST) were estimated using Kaplan-Meier methods. <h3>Results:</h3> A total of N=130 patients were identified (mean age: 52.8 years). At initiation of 2L systemic therapy, over 60% of patients had ECOG score of 0 or 1. Cytotoxic monotherapy was the most frequently prescribed regimen (N=60, 46%) in 2L, followed by combination therapies that included platinum doublet chemotherapy, or bevacizumab doublet or triplet combination therapy (N=45, 35%), pembrolizumab monotherapy (N=19, 15%), and bevacizumab monotherapy (N=6, 5%). Majority of patients initiating 2L had prior bevacizumab exposure in 1L (N=81, 62%), mostly as a triplet or doublet combination therapy. Median OS was 9.1 months (95% confidence interval (CI): 7.2-12.2) after initiation of 2L treatment. Median TTD was 2.8 months (95% CI: 2.5, 3.3), and median TFST was 4.9 months (95% CI: 4.2, 5.7). No statistically significant difference in the outcomes were found when stratified by 2L treatments, although sample sizes were small. <h3>Conclusions:</h3> Diverse treatment patterns were observed in the 2L r/mCC setting. Clinical outcomes in these patients were suboptimal and they did not vary significantly by 2L regimen. Additionally, many patients received combination therapies in 2L after having received similar treatments in 1L, even though there is no evidence to support this practice. These findings demonstrate the unmet medical need for new effective treatment options in this setting.

P-21 Real-world outcomes in patients with advanced or metastatic gastric adenocarcinoma in the United States

Advanced/metastatic gastric cancer is associated with a poor prognosis, often treated with chemotherapy and, more recently, with immune checkpoint inhibitors (ICIs) approved in the USA and Japan. This study aimed to describe the real-world characteristics of patients with advanced/metastatic gastric adenocarcinoma and outcomes following systemic therapy.

Treatment Patterns and Outcomes, Before and After Humulin R U-500 Initiation, Among High-Dose Type 2 Diabetes Mellitus Patients in the United States

ObjectiveSeverely insulin-resistant type 2 diabetes (T2D) patients face unique treatment challenges. Humulin R U-500 (U-500R) as insulin monotherapy with both basal/bolus properties addresses these challenges, although it remains understudied. This retrospective study compared real-world patient characteristics, treatment patterns, and outcomes before and after U-500R initiation. MethodsAdults with T2D on dispensed doses of >180 units/d U-500R monotherapy (index date=first fill) with ≥9-month continuous enrollment both pre- and post-index date and ≥180 units/d insulin pre-index were identified using Veterans Health Administration data (January 1, 2014-January 30, 2017). Overall group was further stratified into elderly and 201 to 300 units dispensed total daily dose (dTDD) subgroups. Syringe and KwikPen users were separately analyzed as subcohorts. Treatment patterns (dTDD), insulin dosage (units/kg), proportion of days covered (PDC) with insulins, and outcomes (HbA1c and hypoglycemic events) were descriptively evaluated, with regression models used to confirm associations between exposure and outcomes. ResultsAmong 951 U-500R initiators (overall group), mean dTDD (248.5 vs 392.1), percentage of patients with insulin dosage >2 units/kg (38.6% vs 88.1%), and mean PDC (73% vs 77%) significantly increased from the pre- to post-index periods (all P<.001). Changes in HbA1c (9.3% vs 8.5%; P<.0001) and hypoglycemia events per patient per year (2.1 vs 3.1, P<.0001) were statistically significant and confirmed by regression models (P<.0001). Subgroups (elderly, 492; 201 to 300 units, 148) and device subcohorts (syringe, 714; KwikPen, 244) showed similar trends. ConclusionU-500R initiation was associated with significantly improved treatment compliance patterns and glycemic control, with modest increase in hypoglycemia events.

Real-world outcomes and clinical characteristics of patients with brain metastases from EGFR mutated non-small cell lung cancer: Data from a large retrospective study (REFLECT).

9086 Background: Brain metastases (BM) frequently occur in patients (pts) with epidermal growth factor receptor mutated non-small cell lung cancer (EGFRm NSCLC) and represent a poor prognostic marker. This study aimed to describe the clinical characteristics, treatment patterns and survival outcomes in EGFRm NSCLC pts treated with 1st or 2nd generation tyrosine-kinases inhibitors (TKIs) in first-line (1L). Methods: The retrospective real-world study REFLECT (NCT04031898) collected data from 896 pts initiating 1L TKI between 1 January 2015-30 June 2018 in Europe and Israel. Descriptive statistics were used to assess demographic and clinical characteristics in subgroups of patients with and without BM. Kaplan-Meier methods were used to estimate median real world progression free survival (mPFS) and overall survival (mOS) from start of 1L. Results: Out of 896 pts, 198 (22.1%) had BM at start of 1L, 134 (15%) developed BM later (any time), and 564 (62.9%) had no sign of BM at the time of data collection. Among pts who later developed BM the median time between the start of 1L and first diagnosis of BM was 13.5 months. Median duration of follow-up was 21.5 months. Of 332 pts with BM at any time 64.2% were female, similar to the ratio in pts without BM (64.0%). At diagnosis, median age was 65 years in pts with BM vs. 70 in those who never developed BM. Of pts with BM at any time, 50.9% had exon 19 deletion, 30.4% L858R point mutation and 18.7 % uncommon EGFR mutations at baseline, compared to 56.6%, 31.7% and 11.7% in pts without BM, respectively. At data collection, 94.9% of the pts with BM at diagnosis had progressed compared to 79.8% among those with no BM. Overall, whole brain radiation was the most frequently used treatment for BM (31.0%) followed by stereotactic radiosurgery (18.1%) and targeted therapies (13.3%). T790M testing rates were highest among pts developing BM later (85.7%) and lowest among those with BM from start (66.1%). The T790M positivity rate was highest in pts developing BM later (65.7%) and lowest among those with BM from start (50.4%). More pts received osimertinib in later lines among those with BM at any time compared to those without BM (51.3% vs 43.8%). Median real world PFS and OS (95% CI) were shorter among pts with BM at baseline compared to those never developing BM: 10.2 (8.8, 11.5) vs 15.2 (13.7, 16.1) months, and 19.4 (17.1, 22.1) vs 30.3 (27.1, 33.8) months, respectively. At the time of data collection, 77.3% of pts with BM at baseline were deceased compared to 52.5% pts with no BM. Conclusions: More than one third of pts included in REFLECT had BM at any time. Uncommon EGFR variants at baseline were observed more frequently in pts with BM. mPFS and mOS were shorter in pts with BM at baseline compared to those never developing BM. These data highlight the need for improved treatment and CNS control in pts with EGFRm NSCLC. Clinical trial information: NCT04031898.

EGFR Exon 20 insertion: Prognostic and predictive values in advanced non-small cell lung cancer, a real-world study.

9062 Background: In Europe, 10-15% of non-squamous non-small cell lung cancer (nsqNSCLC) have EGFR mutations of which 5-12% are an Exon 20 insertion (20ins). Methods: Analysis of Epidemio-Strategy and Medical Economics (ESME) Advanced and Metastatic Lung cancer (AMLC) Data Platform (NCT03848052), a multicenter real-life database using a supervised, retrospective data collection process. The database includes 13737 advanced nsqNSCLC treated from January 2015 at participating centres. The cut-off date for patient follow-up for this analysis was June 30, 2020. The aim of the study was to assess real-world patient characteristics, treatment patterns and clinical outcomes of advanced nsqNSCLC EGFR 20ins. Overall survival (OS) of EGFR cohorts (20ins, 19del/L858R without 20ins, other EGFR mutations) and EGFR wild-type/not tested cohort were assessed. Results: 1549 (11.3%) nsqNSCLC had an EGFR mutation, 61 (3.9%) of whom being an EGFR 20ins. These 61 patients (pts) are mainly female (68.9%), non-smoker (55.7%), with de novo stage IIIB/IV disease (78.6%), PS 0-1 (76.9%). Median age was 68.0 years (q1-q3: 54-74). PD-L1 status was assessed in 34 (55.7%) pts, mainly (n = 20) before first line and 22 (64.7%) had negative result. Most (63.9%) pts had EGFR 20ins positive result available before first line. Almost all pts (95.1%, n = 58) received a systemic therapy with a median number of 3 (q1-q3: 1-4) lines. In first line setting, 74% of the pts received chemotherapy (mainly chemotherapy combination), 13.7% received EGFR TKI (mainly as monotherapy) and 8.6% received immunotherapy only. Median treatment duration for pts treated with CarboPem (n = 19), CisplatinPem (n = 16) and CarboTaxol (n = 6) were 4.7 (q1-q3: 2.6-6.6), 7.4 (q1-q3: 5.0-12.8) and 3.3 (q1-q3: 2.8-3.8) months, respectively. For afatinib (n = 3), erlotinib (n = 2) and gefitinib (n = 1), median treatment durations were 1.6 (q1-q3: 0.5-2.8); 1.8 (q1-q3: 1.4-2.1) and 2.3 months, respectively. After a median follow up of 36.3 (95%CI: 34.1-39.8) months, median OS was 24.3 (95%CI: 19.1-32.6) months; 1 and 2-years OS rates were 82.5% (95%CI: 69.7-90.2) and 52.6% (95%CI: 37.3-65.9), respectively. For pts with 19del/L858R without 20ins (n = 1049) and those with other EGFR mutations (n = 439) median OS were 35.4 (95%CI: 32.6-37.5) and 41.7 (95%CI: 31.9-53.5), respectively compared to 20.7 (95%CI: 20.0-21.8] months for pts EGFR wild type/not tested (n = 12188). Conclusions: This large, national real-world analysis based on medical chart data’s confirm that EGFR 20ins is a rare disease (0.4% of advanced nsqNSCLC). Currently available EGFR TKIs appear to have low efficacy and response to chemotherapy seems identical to that of EGFR wild-type/not tested pts. Prognosis for NSCLC pts with EGFR 20ins mutations was in line with that of EGFR wild type/not tested but worse than common EGFR mutations highlighting the need for advancements for this rare population.

US Real-world Patient Characteristics, Acute Medication Use, and Treatment Patterns for Patients Initiating Fremanezumab (4192)

US Real-world Patient Characteristics, Acute Medication Use, and Treatment Patterns for Patients Initiating Fremanezumab (4192)

Estimated Costs and Long-term Outcomes of Patients With High-Risk Non–Muscle-Invasive Bladder Cancer Treated With Bacillus Calmette-Guérin in the Veterans Affairs Health System

Management of high-risk non-muscle-invasive bladder cancer (NMIBC) represents a clinical challenge due to high failure rates despite prior bacillus Calmette-Guérin (BCG) therapy. To describe real-world patient characteristics, long-term outcomes, and the economic burden in a population with high-risk NMIBC treated with BCG therapy. This retrospective cohort study identified 412 patients with high-risk NMIBC from 63 139 patients diagnosed with bladder cancer who received at least 1 dose of BCG within Department of Veterans Affairs (VA) centers across the US from January 1, 2000, to December 31, 2015. Adequate induction BCG therapy was defined as at least 5 installations, and adequate maintenance BCG therapy was defined as at least 7 installations. Data were analyzed from January 2, 2020, to January 20, 2021. Intravesical BCG therapy, including adequate induction BCG therapy, was defined as at least 5 intravesical instillations of BCG within 70 days from BCG therapy start date. Adequate maintenance BCG therapy was defined as at least 7 installations of BCG within 274 days of the start (the first instillation) of adequate induction BCG therapy (ie, adequate induction BCG plus some form of additional BCG). The Kaplan-Meier method was used to estimate outcomes, including event-free survival. All-cause expenditures were summarized as medians with corresponding interquartile ranges (IQRs) and adjusted to 2019 USD. Of the 412 patients who met inclusion criteria, 335 (81%) were male and 77 (19%) were female, with a median age of 67 (IQR, 61-74) years. Follow-up was 2694 person-years. A total of 392 patients (95%) received adequate induction BCG therapy, and 152 (37%) received adequate BCG therapy. For all patients with high-risk NMIBC, the 10-year progression-free survival rate and disease-specific death rate were 78% and 92%, respectively. Patients with carcinoma in situ (Cis) had worse disease-free survival than those without Cis (hazard ratio [HR], 1.85; 95% CI, 1.34-2.56). Total median costs at 1 year were $29 459 (IQR, $14 991-$52 060); at 2 years, $55 267 (IQR, $28 667-$99 846); and at 5 years, $117 361 (IQR, $59 680-$211 298). Patients with progressive disease had significantly higher median 5-year costs ($232 729 [IQR, $151 321-$341 195] vs $94 879 [IQR, $52 498-$172 631]; P < .001), with outpatient care, pharmacy, and surgery-related costs contributing. Despite adequate induction BCG therapy, only 37% of patients received adequate BCG therapy. Patients with Cis had increased risk of progression, and progression regardless of Cis was associated with significantly increased costs relative to patients without progression. Extrapolating cost figures, regardless of progression, resulted in nationwide costs at 1 year of $373 million for patients diagnosed with high-risk NMIBC in 2019.